Advocacy Group Helps Write Draft FDA Evaluation Guidance on Duchenne Muscular Dystrophy

Patient Group Victory in Muscular Dystrophy Drug Research

In Advocacy by Cecile

Advocacy Group Helps Write Draft FDA Evaluation Guidance on Duchenne Muscular Dystrophy

An American patient advocacy group, Parent Project Muscular Dystrophy, founded by the family members of those afflicted with Duchenne Muscular Dystrophy, has written a draft guidance document for pharmaceutical companies that are or are planning to develop drugs for the condition.

Normnally, these drug development guidances are written and issued by the FDA, detailing how trials should be designed and orchestrated. This time, the draft guidance for Duchenne’s was initiated by the advocacy group. Over 80 parents of sufferers, scientists, pharmaceutical companies and others industry experts helped write the draft, which was then submitted to the FDA in late June 2014.

Robert Temple, the deputy director for clinical science at the FDA’s Center for Drug Evaluation and Research, has said that although the FDA has not commented on when they would issue a response, the FDA is ‘reading it intently, intensively.’

The advocacy group believes that if there is an official FDA guidance on producing drugs for Duchenne, then pharmaceutical companies will be more likely to develop drugs for the condition.

Increasingly, patient advocacy groups are becoming more involved with drug development and research, particularly for rare diseases. Indeed, even US congress has mandated for the FDA to increase patient involvement at all levels.

Pat Furlong, president of the Parent Project and chair of the committee that wrote the draft guidance, decided that the guidance project was important for progress in Duchenne research, particularly in light of recent setbacks in clinical drug trials for Duchenne by Prosensa and PTC. Pat, who has lost two sons to the deadly disease, believes that thing need to be done differently with regards to drug development.

Typically, in clinical trials for Duchenne, the test for a drugs efficacy is a six-minute walking test, which cannot be done by many patients under the age of 7 or those that can no longer walk.

Drugs that are currently in trial phase target some of the specific genetic mutation that cause the disease, a promising start in developing drugs that may one day treat the underlying condition.

In order to speed up drug development, the group believes that other tests beyond the walking test should be used by pharmaceutical companies, as the progressive nature of the disease means many of those afflicted cannot participate in a walking test. Other tests for a drugs efficacy proposed in the draft include the time taken to rise from the floor as well as cardiac measures, pulmonary measures even muscle biopsies.

Whilst member of the advocacy group await a response from the FDA, they are hopeful that their voice will be heard whilst they continue to fight against the fatal disease. 

Read more about it here: A Patients’ Group Scores a Win in Muscular Dystrophy Drug Research